Twelve-year-old girl with rare disease pleads for Ontario to fund life-saving drug that costs $350,000 a year

A 12-year-old girl pleaded Monday for the Ontario government to cover the cost of an expensive drug to fight her rare disease.

Madi Vanstone from Beeton, Ont., has a rare form of cystic fibrosis. The drug Kalydeco has improved her life “significantly,” she said, allowing her to play with her friends and climb the stairs without struggling for breath.

“I didn’t have any energy, I had headaches all the time, bellyaches,” she said. “And now, that’s just gone.”

Her family and friends have been raising money to pay for the drug, which the Progressive Conservatives say costs just under $350,000 a year. But Madi said she wishes they didn’t have to do it.

“If I go off it, I don’t know what I’ll do because this improves my life significantly,” she said.

Her mother, Beth Vanstone, said she’s appealed to the government to pay for the drug, but was told it’s not available because the price is still under negotiation.

The price of Kalydeco has been set in 11 other countries, but here, the price has been negotiated for more than a year, she said.

Premier Kathleen Wynne should help speed things up, Vanstone said.

“There are people who are getting sicker and sicker,” she said. “The wheels have been spinning and we’re not going anywhere.”

People are getting sicker and sicker

Wynne, who met with Madi and her mother Monday, said a group of provinces have been negotiating prices for a number of drugs, including Kalydeco.

Alberta, which is leading those talks, has made three proposals to the manufacturer, Vertex, but it has rejected each one, she said.

It’s not responsible for Ontario to undermine other provinces, Wynne added. But there’s “no question” that the government wants to fund Kalydeco.

“Of course we don’t put a price on a human being’s life,” she told the legislature.

Madi said she was “somewhat” satisfied with her talk with the premier and Health Minister Deb Matthews.

“They were talking about they’re pushing and they’re going to start making it more public and make people know that they’re pushing for this to be done,” she said.